Trial Outcomes & Findings for ESSENCE 2: CyclASol for the Treatment of Signs and Symptoms of Dry Eye Disease (DED) (NCT NCT04523129)

Results Overview

Total Corneal Fluorescein Staining (tCFS) is graded based on the National Eye Institute (NEI) scale. The NEI scale divides the cornea in 5 subregions: central, inferior, superior, nasal, temporal. The score for each region ranges from 0-3; 0 corresponds to no staining (best), 3 corresponds to maximum staining (worst). The total score is the sum of the 5 subregions ranging from 0 (best) - 15 (worst).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

834 participants

Primary outcome timeframe

baseline and 1 month [day 29]

Results posted on

2023-03-10

Participant Flow

Participant milestones

Participant milestones
Measure	CyclASol Ophthalmic Solution Cyclosporine A solution in vehicle CyclASol topical ocular, eye drops: Cyclosporine A solution in vehicle (one drop per eye BID)	Vehicle Ophthalmic Solution Vehicle only Vehicle topical ocular, eye drops: Vehicle (one drop per eye BID)
Overall Study STARTED	423	411
Overall Study COMPLETED	415	402
Overall Study NOT COMPLETED	8	9

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

ESSENCE 2: CyclASol for the Treatment of Signs and Symptoms of Dry Eye Disease (DED)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CyclASol Ophthalmic Solution n=423 Participants Cyclosporine A solution in vehicle CyclASol topical ocular, eye drops: Cyclosporine A solution in vehicle	Vehicle Ophthalmic Solution n=411 Participants Vehicle only Vehicle topical ocular, eye drops: Vehicle	Total n=834 Participants Total of all reporting groups
Age, Continuous	57.6 years STANDARD_DEVIATION 15.36 • n=5 Participants	56.6 years STANDARD_DEVIATION 16.30 • n=7 Participants	57.1 years STANDARD_DEVIATION 15.83 • n=5 Participants
Sex: Female, Male Female	306 Participants n=5 Participants	303 Participants n=7 Participants	609 Participants n=5 Participants
Sex: Female, Male Male	117 Participants n=5 Participants	108 Participants n=7 Participants	225 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	62 Participants n=5 Participants	60 Participants n=7 Participants	122 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	359 Participants n=5 Participants	348 Participants n=7 Participants	707 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Region of Enrollment United States	423 participants n=5 Participants	411 participants n=7 Participants	834 participants n=5 Participants

PRIMARY outcome

Timeframe: baseline and 1 month [day 29]

Total Corneal Fluorescein Staining (tCFS) is graded based on the National Eye Institute (NEI) scale. The NEI scale divides the cornea in 5 subregions: central, inferior, superior, nasal, temporal. The score for each region ranges from 0-3; 0 corresponds to no staining (best), 3 corresponds to maximum staining (worst). The total score is the sum of the 5 subregions ranging from 0 (best) - 15 (worst).

Outcome measures

Outcome measures
Measure	CyclASol Ophthalmic Solution n=409 Participants Cyclosporine A solution in vehicle CyclASol topical ocular, eye drops: Cyclosporine A solution in vehicle	Vehicle Ophthalmic Solution n=395 Participants Vehicle only Vehicle topical ocular, eye drops: Vehicle
Change From Baseline in Total Corneal Fluorescein Staining	-3.96 score on a scale Standard Error 0.146	-3.55 score on a scale Standard Error 0.149

PRIMARY outcome

Timeframe: baseline and 1 month [day 29]

Eye dryness score is rated on a visual analogue scale (VAS) ranging from 0-100; 0 corresponds to no discomfort and 100 to maximum discomfort.

Outcome measures

Outcome measures
Measure	CyclASol Ophthalmic Solution n=409 Participants Cyclosporine A solution in vehicle CyclASol topical ocular, eye drops: Cyclosporine A solution in vehicle	Vehicle Ophthalmic Solution n=395 Participants Vehicle only Vehicle topical ocular, eye drops: Vehicle
Change From Baseline in Eye Dryness Score	-12.2 score on a scale Standard Error 1.29	-13.6 score on a scale Standard Error 1.31

SECONDARY outcome

Timeframe: baseline and 1 month [day 29]

Conjunctival staining was assessed by the investigator in the nasal and temporal sections. The Oxford Scheme was used for each section, with a possible score of 0-5 for each section, where 0=absent and 5=severe. The nasal and temporal scores were summed, for a possible score of 0-10 for each eye.

Outcome measures

Outcome measures
Measure	CyclASol Ophthalmic Solution n=409 Participants Cyclosporine A solution in vehicle CyclASol topical ocular, eye drops: Cyclosporine A solution in vehicle	Vehicle Ophthalmic Solution n=395 Participants Vehicle only Vehicle topical ocular, eye drops: Vehicle
Change From Baseline in Total Conjunctival Lissamine Green Staining	-1.24 score on a scale Standard Error 0.078	-0.91 score on a scale Standard Error 0.079

SECONDARY outcome

Timeframe: baseline and 1 month [day 29]

≥ 1 score improvement for cCFS on National Eye Institute (NEI) scale: Central Corneal Fluorescein Staining (cCFS) is graded based on the NEI scale. The NEI scale divides the cornea in 5 subregions: central, inferior, superior, nasal, temporal. The score for each region ranges from 0-3; 0 corresponds to no staining (best), 3 corresponds to maximum staining (worst).

Outcome measures

Outcome measures
Measure	CyclASol Ophthalmic Solution n=409 Participants Cyclosporine A solution in vehicle CyclASol topical ocular, eye drops: Cyclosporine A solution in vehicle	Vehicle Ophthalmic Solution n=395 Participants Vehicle only Vehicle topical ocular, eye drops: Vehicle
Proportion of Responders in Central Corneal Fluorescein Staining Score	275 Participants	238 Participants

SECONDARY outcome

Timeframe: baseline and 1 month [day 29]

≥ 3 scores improvement for tCFS on National Eye Insititute (NEI) scale: Total Corneal Fluorescein Staining (tCFS) is graded based on the NEI scale. The NEI scale divides the cornea in 5 subregions: central, inferior, superior, nasal, temporal. The score for each region ranges from 0-3; 0 corresponds to no staining (best), 3 corresponds to maximum staining (worst). The total score is the sum of the 5 subregions ranging from 0 (best) -15 (worst).

Outcome measures

Outcome measures
Measure	CyclASol Ophthalmic Solution n=409 Participants Cyclosporine A solution in vehicle CyclASol topical ocular, eye drops: Cyclosporine A solution in vehicle	Vehicle Ophthalmic Solution n=395 Participants Vehicle only Vehicle topical ocular, eye drops: Vehicle
Proportion of Responders in Total Corneal Fluorescein Staining Score	293 Participants	236 Participants

SECONDARY outcome

Timeframe: baseline and 1 month [day 29]

Central Corneal Fluorescein Staining (cCFS) is graded based on the National Eye Institute (NEI) scale. The NEI scale divides the cornea in 5 subregions: central, inferior, superior, nasal, temporal. The score for each region ranges from 0-3; 0 corresponds to no staining (best), 3 corresponds to maximum staining (worst).

Outcome measures

Outcome measures
Measure	CyclASol Ophthalmic Solution n=409 Participants Cyclosporine A solution in vehicle CyclASol topical ocular, eye drops: Cyclosporine A solution in vehicle	Vehicle Ophthalmic Solution n=395 Participants Vehicle only Vehicle topical ocular, eye drops: Vehicle
Change From Baseline in Central Corneal Fluorescein Staining	-0.79 score on a scale Standard Error 0.045	-0.67 score on a scale Standard Error 0.046

SECONDARY outcome

Timeframe: baseline and 2 weeks [day 15]

Total Corneal Fluorescein Staining (tCFS) is graded based on the National Eye Institute (NEI) scale. The NEI scale divides the cornea in 5 subregions: central, inferior, superior, nasal, temporal. The score for each region ranges from 0-3; 0 corresponds to no staining (best), 3 corresponds to maximum staining (worst). The total score is the sum of the 5 subregions ranging from 0 (best) - 15 (worst).

Outcome measures

Outcome measures
Measure	CyclASol Ophthalmic Solution n=418 Participants Cyclosporine A solution in vehicle CyclASol topical ocular, eye drops: Cyclosporine A solution in vehicle	Vehicle Ophthalmic Solution n=403 Participants Vehicle only Vehicle topical ocular, eye drops: Vehicle
Change From Baseline in Total Corneal Fluorescein Staining	-3.53 score on a scale Standard Error 0.144	-2.97 score on a scale Standard Error 0.147

SECONDARY outcome

Timeframe: baseline and 1 month [day 29]

Blurred vision score is rated on a visual analogue scale (VAS) ranging from 0-100; 0 corresponds to no discomfort and 100 to maximum discomfort.

Outcome measures

Outcome measures
Measure	CyclASol Ophthalmic Solution n=409 Participants Cyclosporine A solution in vehicle CyclASol topical ocular, eye drops: Cyclosporine A solution in vehicle	Vehicle Ophthalmic Solution n=395 Participants Vehicle only Vehicle topical ocular, eye drops: Vehicle
Change From Baseline in Visual Analogue Scale (VAS) for Blurred Vision	-7.1 score on a scale Standard Error 1.19	-6.1 score on a scale Standard Error 1.21

Adverse Events

CyclASol Ophthalmic Solution

Serious events: 2 serious events

Other events: 45 other events

Deaths: 0 deaths

Vehicle Ophthalmic Solution

Serious events: 3 serious events

Other events: 37 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	CyclASol Ophthalmic Solution n=423 participants at risk Cyclosporine A solution in vehicle CyclASol topical ocular, eye drops: Cyclosporine A solution in vehicle	Vehicle Ophthalmic Solution n=411 participants at risk Vehicle only Vehicle topical ocular, eye drops: Vehicle
Gastrointestinal disorders Partial Bowel Obstruction	0.00% 0/423 • Adverse event data was were collected over the study period (29 days)	0.24% 1/411 • Number of events 1 • Adverse event data was were collected over the study period (29 days)
Infections and infestations Herpes Simplex Virus	0.00% 0/423 • Adverse event data was were collected over the study period (29 days)	0.24% 1/411 • Number of events 1 • Adverse event data was were collected over the study period (29 days)
Infections and infestations Left Foot Infection	0.24% 1/423 • Number of events 1 • Adverse event data was were collected over the study period (29 days)	0.00% 0/411 • Adverse event data was were collected over the study period (29 days)
Infections and infestations Appendicitis	0.00% 0/423 • Adverse event data was were collected over the study period (29 days)	0.24% 1/411 • Number of events 1 • Adverse event data was were collected over the study period (29 days)
Hepatobiliary disorders Cholelithiasis	0.24% 1/423 • Number of events 1 • Adverse event data was were collected over the study period (29 days)	0.00% 0/411 • Adverse event data was were collected over the study period (29 days)

Other adverse events

Other adverse events
Measure	CyclASol Ophthalmic Solution n=423 participants at risk Cyclosporine A solution in vehicle CyclASol topical ocular, eye drops: Cyclosporine A solution in vehicle	Vehicle Ophthalmic Solution n=411 participants at risk Vehicle only Vehicle topical ocular, eye drops: Vehicle
General disorders Instillation Site Pain	10.6% 45/423 • Adverse event data was were collected over the study period (29 days)	9.0% 37/411 • Adverse event data was were collected over the study period (29 days)

Additional Information

Dr. Sonja Kroesser

Novaliq GmbH

Phone: +49 6221 50259

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60